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Drug & research development » Clinical trials » NCT02107859

Study of Ataluren (PTC124®) in Cystic Fibrosis

Official title: An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124®)

Clinical Trials gov number: NCT02107859

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Purpose: To study the safety and efficacy of ataluren in patients with nonsense mutation CF who participated in the previous Phase 3 study with ataluren (PTC124-GD-009-CF; Study 009).

Phase: 3

Type: Interventional, open-label, single group assignment

Study sponsor: PTC Therapeutics

  • Ability to provide written informed consent (parental/guardian consent and subject assent if <18 years of age)
  • Evidence of completed participation in the double-blind study, PTC124-GD-009-CF (Study 009)
  • Performance of a valid, reproducible spirometry test using the study-specific spirometer during the screening period
  • Confirmed screening laboratory values within the specified central laboratory ranges (hepatic, immune, adrenal, renal, serum electrolytes, and reproductive parameters)
  • In male and female subjects who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and 60-day follow-up period
  • Willingness and ability to comply with all study procedures and assessments, including scheduled visits, drug administration plan, laboratory tests, and study restrictions


Geographical Location
Belgium, France, Israel, Germany, Italy, Spain, Sweden, United States

Number of Participants
51-100 (≥6 years)

Primary Endpoint

Adverse events and laboratory abnormalities over 48 weeks

Secondary Endpoint

At 48 weeks

  • Forced expiratory volume in 1 second (FEV1)
  • Pulmonary exacerbation rate
  • 12 Lead ECG
  • Vital Signs
  • Physical Examination

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  • Experimental: Ataluren (PTC124®)

Oral powder for suspension taken 3 times per day (10-, 10-, and 20-mg/kg morning, midday and evening, respectively) for 48 weeks

View Trial Results